Networks of Centres of Excellence of Canada
Government of Canada

Common menu bar links

Canadian stem cell research leads to groundswell of new clinical trials

Dr. Mehran Anvari and the Image Guided Automated Robot (IGAR)

SCN researchers at the Université de Montréal Institute for Research in Immunology and Cancer have discovered a new molecule that allows for an increase in stem cell transplants. (From left) Jalila Chagraoui, Iman Farès and Guy Sauvageau (Credit: IRIC)

Canadian patients are poised to be among the first globally to receive safe, cutting-edge and rigorously tested stem cell therapies for cancer, Type 1 diabetes, heart disease, stroke and many other chronic illnesses. That’s thanks to a 14-year national initiative to accelerate the clinical use of early-stage research and make Canada the go-to country for patient trials.

It can take several years, if not decades, for a medical breakthrough to move from the lab to the clinical trial stage. So when the Stem Cell Network was launched in 2001, the hope was to move at least one promising therapeutic into patient trials before it sunsetted as a Network of Centres of Excellence (NCE) in 2015.

That goal turned out to be far too modest, underestimating what could be achieved when over 130 biologists, clinicians, bioengineers, lawyers, ethicists and social scientists from universities and hospitals team up with government, industry and international collaborators to move the most cutting-edge research from the lab to the bedside.

Today, in cities across Canada, there are no less than 14 clinical trials underway with another 50 in the pipeline waiting to be funded. Once approved, Canadian patients will be the first to benefit from these revolutionary new treatments.

“We’ve arrived at a tipping point in regenerative medicine where we have many clinical trials that are poised and good to go. That’s something we can all be very proud of. It’s transformative,” says Michael Rudnicki, who joined SCN as scientific director in 2004.

Canada’s climb to the top of the charts in regenerative medicine began in the early 1960s when two Canadians (James Till and Ernest McCulloch) proved the existence of stem cells. That foundational research trained a generation of world-class clinicians and scientists, including Dr. Rudnicki, who also heads the Regenerative Medicine Program and the Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute.

But there were challenges: the community was fragmented, with few opportunities to work collaboratively, particularly across scientific disciplines. SCN transformed this culture by supporting research that encouraged collaboration among talented experts and their trainees from different fields, including bioengineering, medical imaging, bioinformatics, medicinal chemistry, neurology and ethics. The goal was to train a new generation of researchers (over 1,900 to date) and translate fundamental discoveries in stem cell research into clinical applications, commercial products and public policy.

Bringing ethics to the fore

In a first for an NCE, ethical, legal, social and policy issues (ELSI) were integrated into every project SCN funded.

“There was a very high appreciation in the Stem Cell Network from the beginning of ELSI issues, which was unusual for a research network,” says SCN researcher Bartha Knoppers, director of the Centre of Genomics and Policy at McGill University. “Typically, these issues are a by-product of the research, where the attitude used to be ‘give us a consent form and let’s get on with it.’”

Two of the biggest constraints facing stem cell research at the time were ethical concerns and regulatory uncertainty over the use of embryonic stem cells in research. As Dr. Rudnicki points out, at one time, Canada was close “to making all human stem cell research illegal.”

To address this policy vacuum, SCN engaged researchers, legal scholars, ethicists and policymakers from day one to provide credible, research-based evidence and recommendations. The network organized presentations on Parliament Hill, provided expert testimony to House and Senate Standing Committees and participated in over 300 media interviews. Those efforts led Canada to adopt a coherent regulatory and legislative framework for advancing stem cell research and therapies.

Around this same time, scientific advances were finding viable alternatives to the use of embryonic stem cells. Most notable was the discovery by a Japanese research team that induced pluripotent stem cells—generated directly from adult cells—could potentially be used to treat patients. That breakthrough won a Nobel Prize in 2012.

Dr. Knoppers stresses, however, that big ELSI challenges remain, such as stem cell tourism—a small but growing part of the thriving global medical tourism market. Patients desperate for a cure will often pay tens of thousands of dollars for experimental procedures that carry no promise of success, or carry grievous risks of failure.

“Stem cell tourism certainly needs to be addressed. The treatments offered are often unregulated and unproven,” she says. “We need to ensure safety and quality as this research is translated into new therapies. It’s essential not to lose the public trust we have worked so hard to earn.”

SCN’s ELSI research is continuing to open new windows for stem cell research and regenerative medicine—a field that encompasses stem cell research, tissue engineering and gene therapy. In 2013, the Canadian Blood Services launched the country’s first national public bank for umbilical cord blood based in part on a policy recommendation from SCN researchers and ethicists. The resource began receiving donations in Ottawa in September 2013 and expanded to hospitals in Brampton, Edmonton and Vancouver in 2014.

Canada’s leadership in this field is recognized internationally. For example, the Canadian Institutes of Health Research and SCN researchers spearheaded the establishment of the Ethics Working Party of the International Stem Cell Forum. Chaired by Dr. Knoppers and housed at McGill, the working group helps member countries undertake stem cell research within a transparent ethical framework and offers training courses.

From the lab to the bedside

A number of clinical trials are already underway in Canada. In Ottawa and Montréal, researchers are combining gene and cell therapy to stimulate heart repair and heal scarred tissue to improve heart function (Duncan Stewart and Quoc Hung Ly). Another trial, which recently received the go-ahead from Health Canada, will see researchers in Ottawa using bone marrow transplants to improve a patient’s ability to fight infection post-transplant (Duncan Stewart and David Courtman).

Canada is also playing a leading role in a North American trial for stem cell injections for spinal cord injury (Michael Fehlings), and a trial in Edmonton is testing the effectiveness of replacing insulin injections with stem cell implants based on the Edmonton protocol for islet transplantation (James Shapiro and Gregory Korbutt). Meanwhile, a multi-centre trial involving patients in Toronto, Quebec City and Vancouver is testing a technique to expand the number of stem cells that can be generated from a single unit of cord blood, which would overcome the shortage of bone marrow donors and provide hope for more patients with leukemia and other blood-related cancers (Guy Sauvageau).

Other SCN research successes include the development of a STAT3 inhibitor that can effectively kill cancer cells in several types of cancer (Patrick Gunning); the discovery of a switch that turns muscle stem cells into “good” brown fat that could help fight obesity (Dr. Rudnicki’s lab); and success in using cells derived from human embryonic stem cells to reverse Type 1 diabetes in mice (Tim Kieffer).

“We formed a community where no community existed before, so while these projects may have occurred eventually, it would not have happened as quickly as it has,” says Dr. Rudnicki.

Maintaining the momentum

The most commercially promising of SCN’s projects, including Dr. Sauvageau’s success in significantly expanding the number of blood stem cells generated from a single unit of cord blood, are being readied for market by the Centre for Commercialization of Regenerative Medicine (CCRM). Spun off from the SCN as a Centre of Excellence for Commercialization and Research (CECR) in 2011, CCRM’s goal is to overcome two major bottlenecks in bringing regenerative medicine to market: a dearth of early stage capital and access to centralized research infrastructure.

“CCRM was able to launch from a position of strength thanks to the support provided by the Stem Cell Network and MaRS Innovation [another CECR],” says Michael May, CCRM’s CEO and president. “The real successes will come by collaborating and capitalizing on our respective strengths to translate regenerative medicine discoveries into products and therapies.”

Another SCN spin-off, the CellCAN Regenerative Medicine and Cell Therapy Network (a Knowledge Mobilization NCE) is streamlining processes and sharing knowledge between seven cell therapy manufacturing facilities across Canada—all led by SCN researchers. These facilities are providing the capacity to conduct 50-75 new cell-based clinical trials over the next five years.

“We forged an important community that has strategic strength internationally and our research paved the road leading to clinical trials. Both CCRM and CellCAN are very important legacies for the SCN,” says Dr. Rudnicki.

SCN also created and funded the Canadian Stem Cell Foundation, a private-public partnership of researchers, doctors, health charities, business leaders and philanthropists tasked with generating funding for stem cell research and clinical trials. In November 2014, the foundation released a 10-year strategy and action plan that would see Canada become a magnet for more high-quality clinical trials.

“The Stem Cell Network may be sunsetting this year, but our job as a community is far from over,” says Dr. Rudnicki. “We have played a vital role in creating the critical mass of world-class researchers and clinical groups who are prepared to take made-in-Canada discoveries into clinical trials. We can’t afford to lose this momentum.”